NCT05249556

Double-blind, Randomized, Placebo-controlled Trial of Ganaxolone in CDKL5 Deficiency Patients 6 Months to Less Than 2 Years Old

Double-blind, Randomized, Placebo-controlled Trial of Adjunctive Ganaxolone in the Treatment of Seizures Associated With Genetically Confirmed Cyclin-dependent Kinase-like 5 (CDKL5) Deficiency Disorder (CDD) in Pediatric Patients From 6 Months to Less Than 2 Years of Age.

• Status: Not Yet Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: Immedica Pharma AB · Industry
• Sex: All
• Age: 6 Months – 2 Years
• Healthy volunteers: Not accepted

Summary

This study will assess the efficacy, safety, and tolerability of ganaxolone (GNX) compared with placebo (PBO) as adjunctive therapy to the participant's standard anti-epileptic medication for the treatment of seizures in pediatric patients from 6 months to less than 2 years old with genetically confirmed CDD during a 12-week, DB phase. Pharmacokinetic (PK) assessments and population PK analyses will also be performed during this time. The DB phase will be followed by an optional long-term OL phase at which time all participants will receive GNX as an adjunct to their standard anti-seizure medication. Efficacy, safety and tolerability, and PK assessments will continue to be performed.

Conditions

• CDKL5 Deficiency Disorder

Interventions

Type	Name	Description
DRUG	Ganaxolone	Ganaxolone
DRUG	Placebo	Placebo (for ganaxolone)

Timeline

• Start date: 2026-06-01
• Primary completion: 2028-09-01
• Completion: 2028-09-01
• First posted: 2022-02-21
• Last updated: 2025-11-28

Regulatory

• FDA-regulated drug study